A new report from the U.S. Food and Drug Administration (FDA) notes that the Center for Drug Evaluation and Research (CDER) approved 53 novel drugs last year. These figures are in addition to innovative medicines that are approved through FDA’s Center for Biologics Evaluation and Research—which include for example, cell therapies, gene therapies, vaccines and other biologic medicines.
Of the CDER approvals, 22 (42%) were designated breakthrough therapies for serious, life-threatening disease and 21 (39%) were considered first-in-class treatments, meaning according to CDER they “often have mechanisms of action different from those of existing therapies.” While medicine approval numbers ebb and flow each year, 2020 saw exciting advancements in drug research and development despite unprecedented challenges with the COVID-19 pandemic. These approvals represent the unwavering commitment of biopharmaceutical researchers to developing treatments for patients with range of complex and challenging diseases.
Beyond the novel drugs that became available to patients for the first time in 2020, the report also highlights the tremendous innovation provided by new and expanded uses of existing medicines for use in new diseases or treatment populations, as well as new formulations and dosage forms.
Here are four key takeaways from CDER’s annual report:
Improved Patient Access for Americans
40 of the 53 novel therapies (75%) approved in 2020 were approved by the FDA before receiving regulatory approval in another country, solidifying our nation’s role as the global leader in biopharmaceutical innovation. Additionally, nearly all novel medicines (92%) were approved on the “first cycle” of review, meaning these candidate therapies were not delayed by re-submission.
Continued Progress for Rare Diseases
Among last year’s approvals were 31 new therapies for rare diseases. These new medicines include treatments for devastating neurodegenerative diseases such as spinal muscular atrophy and Duchenne muscular dystrophy, dangerous inflammatory conditions such as hereditary angioedema, and several rare and deadly forms of cancers. Many of these provide first-time treatments for patients who previously had few or no medicines available to treat their conditions, while others offer new treatment options for patients and better management of disease.
Rare diseases are defined as affecting fewer than 200,000 American patients, but collectively these diseases impact 1 in 10 Americans. Today, there remains tremendous unmet need as only 5% of rare diseases have available treatment options. This past year’s progress builds on the significant advances reported for 2019 and continues to expand the number of treatments available to the 30 million Americans affected by rare diseases.
Infectious Disease Innovation
Among last year’s approvals was the first U.S. treatment for COVID-19 patients. 2020 also saw continued progress against HIV with a first-in-class therapy for patients living with HIV who cannot manage the disease with available antiretroviral medications due to resistance, intolerance or safety considerations. Two treatments for Ebola infection and severe Malaria which previously lacked treatment options in the United States also became available to patients in 2020.
Advances in New / Expanded Uses and New Dosage Forms or Formulations of Previously Approved Medicines Provide Critical Treatment Options to Patients
In 2020, CDER approved new uses of several cancer medicines to treat different forms of cancer, including bladder cancer, colorectal cancer, kidney cancer, prostate cancer, lung cancer and malignant pleural mesothelioma. Likewise, many previously approved medicines, after further research and development, were expanded to broader cancer treatment populations.
In addition to new cancer treatment options, a medicine originally approved to treat a form of leukemia was subsequently approved to treat relapsing forms of multiple sclerosis. A new injectable formulation of a previously available treatment for multiple myeloma was also approved, cutting the time to administer the medicine from several hours to just a few minutes.
Three previously approved diabetes medicines were also approved for patients with chronic heart failure and to help reduce the risk of major adverse cardiovascular events.
CDER also approved new uses for a previously approved medicine for patients with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia, importantly expanding the arsenal of medicines available to battle these infections. Additionally, a new oral formulation of an already approved HIV medicine was approved to treat pediatric patients at least 4 weeks old in combination with other antiretroviral treatments, helping to ensure earlier treatment in pediatric HIV patients in whom the disease progresses more quickly than adults.
Approvals from CDER demonstrate the incredible progress made by U.S. biopharmaceutical companies during an unprecedented year. Moreover, in 2020 CDER met its Prescription Drug User Fee Act goal dates for 100% of the novel drugs approved, signaling an efficient and effective review process.
As we look ahead to 2021, we are excited to build on these great strides and bring new innovative solutions to patients in need. While the COVID-19 pandemic continues to significantly impact on our nation’s health care system, the innovative biopharmaceutical industry continues to work around the clock to bring new treatments and vaccines for patients. Today, one treatment has been approved and five treatments and two vaccines have already received emergency use authorization to address the COVID-19 pandemic and more are expected.
Learn more about last year’s advancements by reading the CDER FDA New Drug Approval Report.
